FDA授予LP-184治疗多形性胶质母细胞瘤和恶性胶质瘤的孤儿药称号

Glioblastoma (GBM) is the most common primary cancer of the brain, a type of glioma, and the most common malignant tumor in neurosurgery. Located in the central nervous system, most of them grow in various parts of the supratentorial cerebral hemisphere, and the most common location is the frontal lobe.

Lantern Pharma announced that the FDA has granted orphan drug designation to LP-184 as a potential treatment option for patients with glioblastoma multiforme and other malignant gliomas.

LP-184 is a drug that acts on DNA repair pathways, namely the nucleotide excision repair (NER) and homologous recombination (HR) pathways. Previous analyzes have demonstrated potential benefit from LP-184 in patients with glioblastoma multiforme, elevated expression of PTGR1, and defects in DNA damage repair components.

In preclinical studies conducted in collaboration with Johns Hopkins University, LP-184 was found to inhibit tumor growth by more than 106% in two subcutaneous xenograft models of glioblastoma multiforme. The formulation also prolonged the survival of mice with an intracranially implanted tumor model (U87) compared with mice that did not receive any drug.

When LP-184 was injected intravenously for 2 cycles, the drug reduced the volume of subcutaneous xenograft tumors in mice by more than 85%. In a mouse orthotopic glioblastoma xenograft tumor model, the median overall survival after a single cycle of treatment was 42 days, which was a statistically significant extension compared with the control group (33 days).

"We believe LP-184's ability to cross the blood-brain barrier, its anti-tumor efficacy, and its sensitivity to relevant biomarkers could make it a lifesaver for patients with glioblastoma multiforme and other aggressive central nervous system tumors," said Panna Sharma, president and CEO of Lantern Pharma. "LP-184 can be used either as a monotherapy or as a synergist in combination with other drugs."

Previously, in August 2021, the FDA granted LP-184 orphan drug designation for the treatment of patients with pancreatic cancer. Preclinical data shows that it can significantly and rapidly shrink tumors in in vivo mouse models by more than 90% within 8 weeks. Over the same period, tumors in untreated mice grew 11-fold.

The drug showed activity in six pancreatic cancer cell lines and five patient-derived xenograft in vitro tumor models. Across all cell lines and models, LP-184 significantly reduced cancer cell numbers and slowed cancer cell growth.

In summary, LP-184 can significantly reduce the growth rate of cancer cells. We expect this drug to achieve good efficacy in clinical trials and provide new treatment options for more patients.

References:

https://www.onclive.com/view/fda-grants-orphan-drug-designation-to-lp-184-for-glioblastoma-multiforme-and-malignant-gliomas